Scholar Rock aims for a 2025 launch of apitegromab in SMA, expecting $2 billion in global revenue and significant growth from upcoming trials and favorable data.

Creatinine, a byproduct of muscle function, may be more sensitive in tracking SMA progression and response to treatment than ...

While investors and analysts push for a deal, Biogen CEO Chris Viehbacher and Head of Development Priya Singhal refuse to ...

Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene ...

Advocates rallied outside the State House on Tuesday for special education funding.The group urged lawmakers to allocate more ...

Known online as Squirmy and Grubs, YouTubers Hannah and Shane Burcaw have over 1.8 million subscribers. The married ...

John Hood, CEO, Endeavor BioMedicines discusses partnering with advocacy groups to increase awareness of clinical trials for idiopathic pulmonary fibrosis.

REGENXBIO to receive $110 million upfront, potential milestone payments of up to $700 million and meaningful double- ...

NORTH CHICAGO, IL and ROCKVILLE, MD, USA I January 13, 2025 I AbbVie (NYSE: ABBV) and REGENXBIO Inc. (Nasdaq: RGNX) today announced updates to the ...

Crystal Rondeau, who has spinal muscular atrophy Type 2 and which has left her with only one finger to press an alarm button ...

To achieve the goals set out in its ‘Vision 2030’, argenx has set the following priorities for 2025: Expand the global VYVGART opportunity by reaching more patients broadly across MG, CIDP and ITP ...

Crystal Rondeau’s muscle wasting disease has left her with only one finger to press an alarm button or send a text. The ...